Adamson J W, Schuster M, Allen S, Haley N R
New York Blood Center, N.Y.
Acta Haematol. 1992;87 Suppl 1:20-4. doi: 10.1159/000204784.
In an attempt to determine predictors of response to recombinant human erythropoietin (r-HuEPO) therapy in 20 patients with various subtypes of myelodysplastic syndrome (MDS), plasma concentrations of transferrin receptor protein were measured before and after 4 doses of r-HuEPO. An r-HuEPO dosage of 150 U/kg was administered subcutaneously 3 times weekly and increased to 300 U/kg in patients who failed to raise plasma concentrations of transferrin receptor protein by at least one third. Ten (50%) patients had an effective clinical response to therapy by reducing (greater than 50%) or eliminating transfusion requirements, or by showing an improvement in haematocrit of greater than or equal to 6 percentage points. Changes in plasma transferrin receptor protein concentrations failed to predict which patients would eventually respond to r-HuEPO therapy. A subset of MDS patients demonstrated a delayed response to therapy in order to achieve a satisfactory clinical outcome. Precise predictors of response, either laboratory or clinical, remain to be determined. Continued research is warranted in this group of patients in order to specifically target r-HuEPO therapy. It is, however, likely that r-HuEPO therapy will have an effective and important role in this subset of MDS patients.
为了确定20例不同亚型骨髓增生异常综合征（MDS）患者对重组人促红细胞生成素（r-HuEPO）治疗的反应预测指标，在4剂r-HuEPO治疗前后测量了转铁蛋白受体蛋白的血浆浓度。r-HuEPO剂量为150 U/kg，每周皮下注射3次，对于未能使转铁蛋白受体蛋白血浆浓度至少提高三分之一的患者，剂量增加至300 U/kg。10例（50%）患者通过减少（大于50%）或消除输血需求，或血细胞比容提高大于或等于6个百分点，对治疗有有效的临床反应。血浆转铁蛋白受体蛋白浓度的变化未能预测哪些患者最终会对r-HuEPO治疗产生反应。一部分MDS患者为了获得满意的临床结果，对治疗表现出延迟反应。无论是实验室指标还是临床指标，反应的精确预测指标仍有待确定。有必要对这组患者继续进行研究，以便专门针对r-HuEPO治疗。然而，r-HuEPO治疗很可能在这部分MDS患者中发挥有效且重要的作用。
